PASSINI, MARCO, A.,ZIEGLER, ROBIN, J.,DODGE, JAMES,SHIHABUDDIN, LAMYA,CHENG, SENG, H.
申请号:
EP07763560
公开号:
EP1986661A4
申请日:
2007.02.08
申请国别(地区):
EP
年份:
2012
代理人:
摘要:
This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).